EU/3/21/2410: Orphan designation for the treatment of medullary thyroid carcinoma

2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib)

Table of contents

Overview

This medicine was designated as an orphan medicine for the treatment of medullary thyroid carcinoma in the European Union on 19 February 2021.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Key facts

Active substance
2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib)
Intended use
Treatment of medullary thyroid carcinoma
Orphan designation status
Positive
EU designation number
EU/3/21/2410
Date of designation
19/02/2021
Sponsor

Propharma Group The Netherlands B.V.
Schipholweg 73
2316 ZL Leiden
Zuid-Holland
Netherlands
E-mail: info@propharmagroup.com

Update history

DateUpdate
September 2023The sponsorship was transferred from Southwood Research Ltd., Netherlands to Propharma Group The Netherlands B.V., Netherlands.

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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